Highlights
Rarely has a new technology so squarely lent itself to one disease. Here’s why.
Major biotechs, including Beam, Wave Life Sciences, Korro Bio, Tessera, and Yoltech are targeting a single misspelled letter of DNA.
Analysts see a multibillion-dollar potential market.
Biotechs are spending billions to cure a rare liver disorder most Americans have never heard of. The contentious race features dueling technologies, patent wars, a broken alliance, and a boiling competition between the U.S. and Chinese drug industries.
The disease, known as alpha-1 antitrypsin deficiency (AATD), is a slow-moving disaster for patients. Thanks to a single misspelled letter of DNA, their livers produce a mutant version of a protein that normally travels through the bloodstream and protects the lung from damage.
This article is exclusive to STAT+ subscribers
Unlock this article — plus in-depth analysis, newsletters, premium events, and news alerts.
Already have an account? Log in
View All Plans
To read the rest of this story subscribe to STAT+.
Subscribe